Applying the Evidence-Generation Continuum Concept to Support Patient Access to Innovative Therapies in France: A Use Case in Rare Diseases
Author(s)
Nassim Kamar, MD1, Catherine Cordonnier, MD2, Faouzi Saliba, MD3, Cinira Lefevre, PhD4, Michele Maric, MSc5, Sophie Alain, MD6.
1IFR-BMT, University Paul Sabatier, CHU Rangueil and Inserm U1043, Toulouse, France, 2Assistance Publique – Hopitaux de Paris (AP-HP), Henri Mondor Hospital and University Paris-Est-Créteil, Créteil, France, 3CHU Lille, Lille, France, 4Patient Value & Access, TAKEDA France, Paris, France, 5Medical Affairs, TAKEDA France, Paris, France, 6Limoges University Hospital and UMR Inserm 1092, University of Limoges, Limoges, France.
1IFR-BMT, University Paul Sabatier, CHU Rangueil and Inserm U1043, Toulouse, France, 2Assistance Publique – Hopitaux de Paris (AP-HP), Henri Mondor Hospital and University Paris-Est-Créteil, Créteil, France, 3CHU Lille, Lille, France, 4Patient Value & Access, TAKEDA France, Paris, France, 5Medical Affairs, TAKEDA France, Paris, France, 6Limoges University Hospital and UMR Inserm 1092, University of Limoges, Limoges, France.
OBJECTIVES: Randomized controlled clinical trials are the gold standard for French Health Technology Assessments, and real-world evidence has been considered as complementary/supportive data to strengthen value demonstration. The evidence generation continuum (EGC) concept has been deployed in France for maribavir (indicated for treating CMV infection/disease refractory to prior therapies in transplanted patients): alongside providing early treatment access for patients in high unmet medical need, the early access program (AP2), part of maribavir EGC, allowed to generate data complementary to the SOLSTICE trial. The purpose of this work is to describe the EGC concept and describe the data generated on maribavir as part of the AP2.
METHODS: Prospective longitudinal data were collected from patients treated with maribavir across 117 centers in France. Patients were followed from maribavir treatment initiation to the end of treatment. Patient characteristics were described at maribavir initiation using descriptive statistics, including frequency and percentages were reported. Treatment duration was analyzed using the Kaplan-Meier estimator to describe median time to treatment discontinuation and respective 95% CIs.and treatment duration.
RESULTS: Overall, 347 patients were included in the AP2, with patient characteristics consistent with those of patients treated in the pivotal trial (median age of 58 years, majority of males [62.5%] and patients who had solid organ transplants [85.6%]). Among 361 patients with maribavir treatment, 70% had a completed end of treatment form available at the end of the AP2 program, with a median treatment duration of 57 days (57-59), aligned with the 8-week recommended duration in the maribavir SmPC.
CONCLUSIONS: The EGC applied to maribavir supported its value demonstration, while allowing for patient access in a context of high unmet medical need. These data were complementary to those from the pivotal trial and confirmed existing data from French maribavir users.
METHODS: Prospective longitudinal data were collected from patients treated with maribavir across 117 centers in France. Patients were followed from maribavir treatment initiation to the end of treatment. Patient characteristics were described at maribavir initiation using descriptive statistics, including frequency and percentages were reported. Treatment duration was analyzed using the Kaplan-Meier estimator to describe median time to treatment discontinuation and respective 95% CIs.and treatment duration.
RESULTS: Overall, 347 patients were included in the AP2, with patient characteristics consistent with those of patients treated in the pivotal trial (median age of 58 years, majority of males [62.5%] and patients who had solid organ transplants [85.6%]). Among 361 patients with maribavir treatment, 70% had a completed end of treatment form available at the end of the AP2 program, with a median treatment duration of 57 days (57-59), aligned with the 8-week recommended duration in the maribavir SmPC.
CONCLUSIONS: The EGC applied to maribavir supported its value demonstration, while allowing for patient access in a context of high unmet medical need. These data were complementary to those from the pivotal trial and confirmed existing data from French maribavir users.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA37
Topic
Clinical Outcomes, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Rare & Orphan Diseases